PTC Therapeutics, Inc announced an exclusive research collaboration and licensing agreement with Pfizer Inc for the development of pharmaceuticals through the application of PTC's GEMS (Gene Expression Modulation by Small-Molecules) technology.

Under the terms of the agreement Pfizer has made an initial upfront payment of $10 million, will purchase an equity stake of $10 million subject to certain closing conditions, and provide supportive funding for PTC's research efforts.

At Pfizer's option, the collaboration may include up to 10 targets and PTC could earn up to $121 million in milestones per target based on the achievement of certain development, regulatory, and commercial goals. Pfizer will receive exclusive worldwide rights and pay PTC royalties on worldwide net sales of any products resulting from this collaboration.

"We are very pleased with Pfizer's significant commitment to our scientific approach," said Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics. "We have demonstrated the potential of the GEMS technology through our multiple internal programs and collaborations based on GEMS and believe this collaboration with Pfizer will broaden the applicability of the technology into new areas of high unmet medical need."

GEMS is PTC's novel and proprietary technology platform for the identification of small-molecules that modulate post-transcriptional control mechanisms. PTC has successfully employed the GEMS technology in drug discovery programs in oncology, infectious diseases, cardiovascular diseases, and neuromuscular disorders. PTC's most advanced compound identified through the GEMS technology is PTC299, an orally-administered small molecule designed to inhibit the production of vascular endothelial growth factor (VEGF), which functions through a mechanism that is distinct from other VEGF inhibitors. PTC299 is currently in phase 1 clinical trials.